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QTORINTM rapamycin 3.9% for Gorlin Syndrome

QTORIN™ rapamycin 3.9%is a novel targeted topical therapy being studied for Gorlin syndrome. Palvella is executing a phase 2b clinical study (CODY) for people with Gorlin syndrome.

About Gorlin Syndrome: A lifelong risk of malignant skin cancer

Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome, is a serious rare life-long genetic disease caused by a mutation in the PTCH1 gene leading to abherrant hedgehog signaling. Recurrent basal cell carcinomas (BCCs) are a hallmark of this disease.  Individuals with Gorlin syndrome can develop hundreds or even thousands of Basal Cell Carcinomas (BCCs), oftentimes beginning in childhood. BCCs are a malignant skin cancer requiring repeated and potentially disfiguring surgical removal for patients with Gorlin syndrome.  Gorlin syndrome affects an estimated 11,000 people in the United States.

There are no FDA approved therapies for Gorlin syndrome.

Targeting Gorlin syndrome

Palvella initiated internal research efforts on the potential for QTORINTM rapamycin 3.9% in Gorlin syndrome in 2017. QTORIN™ rapamycin 3.9%  is a novel targeted topical therapy designed to inhibit mTOR.  The mTOR pathway has been shown as a major driver of BCC tumorigenesis in scientific research¹,2. This approach aims to avoid some of the limitations of the direct hedgehog inhibitors, such as resistance and recurrence. Palvella is executing a phase 2b clinical study investigating the potential of QTORIN™ rapamycin 3.9% for the prevention of Basal Cell Carcinomas associated with Gorlin syndrome.

Hua, H., Kong, Q., Zhang, H. et al. Targeting mTOR for cancer therapy. J Hematol Oncol 12, 71 (2019) doi:10.1186/s13045-019-0754-1

Kim, A., Back, J. et al. SOX9 Transcriptionally Regulates mTOR-Induced Proliferation of Basal Cell Carcinomas. J Invest Dermatol. 2018 August; 138(8): 1716–1725.

Clinical Study is Gorlin syndrome is now enrolling!

Palvella is enrolling a phase 2b clinical study for the prevention of BCCs in patients with Gorlin syndrome! Please use the link below to learn more about this study.

Anticipated Requirements Include:
  • Confirmed Gorlin syndrome
  • Adults, age 18 or older
  • History of BCCs on the face
  • Cannot have participated in another Gorlin clinical study in past 3 months

Resources for Gorlin

Gorlin Syndrome Alliance

Founded in 2000, the Gorlin Syndrome Alliance is the primary patient advocacy group for Gorlin Syndome in the U.S. Their mission is to thoughtfully support, comprehensively educate and aggressively seek treatments and a cure for Gorlin Syndrome, its manifestations, and BCCs.

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National Organization for Rare Disorders

NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 280 patient organizations members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.

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